[Single-center clinical study of upadacitinib in children with refractory inflammatory bowel disease].

内容摘要

To study the efficacy and safety of upadacitinib in children with refractory inflammatory bowel disease (IBD). Clinical data of eight children with refractory IBD treated with upadacitinib at the Children's Hospital, Zhejiang University School of Medicine, from December 1, 2023 to May 20, 2025 were retrospectively collected to evaluate efficacy and safety. Eight children were included [Crohn's disease (CD), n=5; ulcerative colitis (UC), n=3]; seven were male and one was female. At baseline, six patients had mild disease activity (CD, n=4; UC, n=2) and two had severe activity (CD, n=1; UC, n=1). In UC, all three patients achieved clinical remission by week 2; the week-8 steroid-free clinical remission rate was 67% (2/3). In CD, after 4 weeks of upadacitinib, one patient achieved a clinical response and two switched to other drugs due to persistent activity; the week-12 clinical response rate was 20% (1/5). After 12 weeks, one additional patient switched therapy due to disease activity, and two received combination therapy with thalidomide and exclusive enteral nutrition, respectively. The median follow-up duration was 28 weeks. Overall, five patients achieved clinical remission; among CD, two achieved clinical remission at weeks 14 and 20, respectively. Among eight patients, one episode of norovirus infection occurred, complicated by hypovolemic shock. Upadacitinib can induce a rapid response in children with refractory UC. In refractory CD, combining upadacitinib with agents of different mechanisms may increase the clinical remission rate. Multicenter studies with larger samples are required for confirmation, and vigilance for infection during treatment is warranted. 目的: 分析乌帕替尼治疗难治性炎症性肠病（inflammatory bowel disease, IBD）患儿的有效性及安全性。方法: 回顾性收集2023年12月1日—2025年5月20日在浙江大学医学院附属儿童医院接受乌帕替尼治疗的8例难治性炎症性肠病（inflammatory bowel disease, IBD）患儿的临床资料，评估乌帕替尼对难治性IBD患儿的有效性及安全性。结果: 8例患儿中，克罗恩病（Crohn's disease, CD）5例，溃疡性结肠炎（ulcerative colitis, UC）3例。男7例，女1例。在基线水平，疾病轻度活动期6例（CD 4例，UC 2例），重度活动期2例（CD 1例，UC 1例）。3例UC患儿经乌帕替尼治疗2周均获得临床缓解，8周无激素临床缓解率67%（2/3）;5例CD患儿经乌帕替尼治疗4周后，1例获得临床应答，2例因疾病活动更换为其他药物，12周临床应答率为20%（1/5），12周后1例因疾病活动更换药物，另2例分别联合沙利度胺及全肠内营养治疗。随访时长中位数为28周。最终5例患儿获得临床缓解，2例CD患儿分别于第14周及第20周获临床缓解。8例患儿共发生1例次诺如病毒感染，合并低血容量性休克。结论: 乌帕替尼对难治性UC患儿可快速起效，对难治性CD患儿，乌帕替尼联合不同作用机制的药物治疗可增加临床缓解率，仍需要多中心、大样本的研究进一步证实，治疗过程中需警惕感染的风险。.